Question

Answer the following question.
Two children, A and B aged 4 and 5 years respectively visited a hospital with a similar genetic disorder. The girl A was provided enzyme-replacement therapy and was advised to revisit periodically for further treatment. The girl, B was, however, given a therapy that did not require revisit for further treatment.

How was the girl B cured permanently?

Answer 1

Girl B was treated with gene therapy through the gene isolated from bone marrow cells producing ADA is introduced into cells at early embryonic stages which are a permanent cure so the patient is cured permanently.